prima applicazione della terapia genica sull'UOMO
I think we have reached a turning point ...
soon (I hope) a comment is a bit ' more personal.
gene therapy to treat Leber congenital dell'amaurosi
The New England Journal of Medicine have published the results of the first intervention gene therapy on ' man to treat Leber congenital amaurosis, a severe form of hereditary blindness.
The intervention was successful and is the result of a long and complex work of an international team that made the part of the Children's Hospital of Philadelphia and Naples, Telethon Institute of Genetics and Medicine (Tigem) and the Department of Ophthalmology of the Second University of Naples.
The Leber congenital amaurosis is a form of retinopathy of genetic origin and cause blindness or serious eye injury in the early months of life.
currently no cure, but gene therapy could be the right strategy to cure or at least against this disease.
During the clinical trial, doctors have injected into the subretinal space of the eye a viral vector containing a correct copy of the altered gene that causes the disease . The normal gene, it was plugged in the retina and produced the missing protein in individuals malati.I results been very positive. For some months after the treatment, all patients were observed significant improvements in visual function : improved retinal response to light, the ability to run tests of mobility and the perception of the visual field.
Moreover, the treatment did not cause toxic effects. Participated in this first clinical trial of a girl 19 years of Pavia and twins 26 years of Agrigento.
The Italian part of the research (funded by the Telethon Foundation) was coordinated by Alberto Auricchio, Tigem researcher and associate professor at the University Federico II of Naples. Also for the Tigem, have also collaborated Enrico Maria Surace and Sandro Banfi, which dealt with genetic screening of patients eligible for intervention.
The part about the recruitment and follow-up of patients were coordinated by Francesca Simonelli Department of Ophthalmology of the Second University of Naples, while the synthesis of viral vector and the surgery took place at Children's Hospital Philadelphia, under the supervision of Jean Bennett .
These results were presented in Rome by Alberto Auricchio and Francesca Simonelli, the presence of Andrea Ballabio (Tigem Director) and Susanna Agnelli (President of the Telethon). Now researchers
procederanno col testare l'assenza di tossicità anche a dosaggi più alti e su un numero maggiore di pazienti e valutando gli effetti a lungo termine .
La terapia genetica dimostra di essere efficace e destinata a migliorarsi sempre più e, con la recente scoperta di vettori in grado di trasferire nell'organismo geni di grandi dimensioni, si candida ad essere sfruttata anche per altre malattie causate dall'alterazione di geni di grandi dimensioni come la fibrosi cistica, la malattia di Stargardt, la distrofia muscolare di Duchenne e l'emofilia di tipo A.
Redazione MolecularLab.it (29/04/2008)
Wednesday, April 30, 2008
Tuesday, April 8, 2008
Does Birth Control And Aging?
I CARE
vuole 200 parole prof?
.. no way, at least on this occasion no ..
thanks for the opportunity!
many feelings on this morning out of the ordinary ..
WONDER - well yes .. I was not ready for something new, perhaps I was expecting the usual ... and leave the conference scontatezza is always something good that gives you energy! a breath of fresh air to receive the testimony of a different experience, of how to integrate our study with something more.
feel CHARGE - I can not give up me to believe that this may cause you to close your wings and fly no more, I have too much energy into why this happens .. I made this choice not to learn how to repair the machines, but because I am convinced that man is a machine and that medicine can teach us much about man and humanity, the value of life and of death. This morning all my enthusiasm has done it again peeps ... I talked about ways to go in a new way to search for more stimulus of interest to cultivate. Ma ..
ANXIETY - which the road? What should I spend? I need a goal. Need to fight for something, my battle where you'll spend all the energy that I feel I have .. I have said that the time of living. But wait, is the most difficult you can ask for a life.
MEETING - beyond all, as always, to weave the plot is the meeting with people .. that makes the heart explode by how beautiful it is. difficult to explain the enthusiasm that I feel to read something over their faces, to realize a piece of life ... wonderful mechanisms of life.
I CARE.
What to add to this? Maybe we need to say anything more.
It 's my rule and my needs. My cross and my delight.
But I'm proud.
vuole 200 parole prof?
.. no way, at least on this occasion no ..
thanks for the opportunity!
many feelings on this morning out of the ordinary ..
WONDER - well yes .. I was not ready for something new, perhaps I was expecting the usual ... and leave the conference scontatezza is always something good that gives you energy! a breath of fresh air to receive the testimony of a different experience, of how to integrate our study with something more.
feel CHARGE - I can not give up me to believe that this may cause you to close your wings and fly no more, I have too much energy into why this happens .. I made this choice not to learn how to repair the machines, but because I am convinced that man is a machine and that medicine can teach us much about man and humanity, the value of life and of death. This morning all my enthusiasm has done it again peeps ... I talked about ways to go in a new way to search for more stimulus of interest to cultivate. Ma ..
ANXIETY - which the road? What should I spend? I need a goal. Need to fight for something, my battle where you'll spend all the energy that I feel I have .. I have said that the time of living. But wait, is the most difficult you can ask for a life.
MEETING - beyond all, as always, to weave the plot is the meeting with people .. that makes the heart explode by how beautiful it is. difficult to explain the enthusiasm that I feel to read something over their faces, to realize a piece of life ... wonderful mechanisms of life.
I CARE.
What to add to this? Maybe we need to say anything more.
It 's my rule and my needs. My cross and my delight.
But I'm proud.
Wednesday, April 2, 2008
Build Your Own Desktop Hutch
contesto
not wanting in my previous post I brought my own assessment of the complexity, which I think is well suited to the discussion on the "value of context."
in high school I hated physics, because I have taught as pure concept.
math so much, I liked because I did well ... even if no teacher was able to satisfy my doubts about the usefulness of mathematics, leaving me in limbo without context of fun ..
launch a proposal: why do not we put up a series of videos which explain and contextualize the formulas of physics, in the footsteps of Lee Lefever?
might be interesting ... I definitely would take to recover a lost part of human knowledge;)
not wanting in my previous post I brought my own assessment of the complexity, which I think is well suited to the discussion on the "value of context."
in high school I hated physics, because I have taught as pure concept.
math so much, I liked because I did well ... even if no teacher was able to satisfy my doubts about the usefulness of mathematics, leaving me in limbo without context of fun ..
launch a proposal: why do not we put up a series of videos which explain and contextualize the formulas of physics, in the footsteps of Lee Lefever?
might be interesting ... I definitely would take to recover a lost part of human knowledge;)
Tuesday, April 1, 2008
Why Do You Use The Internet
complessità
Imagine the old hall of a university department, a classroom but a bit 'special ... a large room with tables with gray marble board rialzato; alle pareti scaffali in legno con ante in vetro chiusi a chiave, armadi, attaccapanni, lavagne.
In realtà è un'aula speciale: ci si pratica(va?)no le dissezioni.
Sugli scaffali con le ante in vetro si vede ogni genere di organo o pezzo di corpo umano, all'occorrenza credo mummificato a dovere.
A noi studenti del XXI secolo non è concesso praticare dissezioni, ma i nostri professori hanno imparato qui.
Questa aula la usiamo per le esercitazioni di anatomia; ci lasciano qualche osso (vero) sui tavoli e noi ci giochiamo, li guardiamo e studiamo, ce li imprimiamo nella mente.
Ero ad una di queste esercitazioni; a tempo scaduto eravamo rimasti in pochi, e un gruppetto di sole donne temerarie (Including me) to encircle the prof and his laptop. Someone asks what kind of material can be found on the Internet, prof says "everything" and to demonstrate, as a movie, it pulls all its various videos of dissections.
Crazy.
Not so much disgust: the impression is relative, for those not afraid of blood, bone, fat.
But at the moral and human is devastating.
see a body, a few days before was still a living person with a life and history, reduced to a mere heap of flesh, no dignity.
find yourself making these thoughts ... yes man - no man ... yes dignity - no dignity.
children, is tough, and quite a lot. This right is tough, because he wants to get you to the study of microscopic and in the meantime is likely to get away from the big picture.
I'll have to go through the dissection through the bodies of dead and lifeless, sooner or later.
But I promise to never lose the overview. The complexity as something more than the sum of its parts.
The feeling of humanity.
Imagine the old hall of a university department, a classroom but a bit 'special ... a large room with tables with gray marble board rialzato; alle pareti scaffali in legno con ante in vetro chiusi a chiave, armadi, attaccapanni, lavagne.
In realtà è un'aula speciale: ci si pratica(va?)no le dissezioni.
Sugli scaffali con le ante in vetro si vede ogni genere di organo o pezzo di corpo umano, all'occorrenza credo mummificato a dovere.
A noi studenti del XXI secolo non è concesso praticare dissezioni, ma i nostri professori hanno imparato qui.
Questa aula la usiamo per le esercitazioni di anatomia; ci lasciano qualche osso (vero) sui tavoli e noi ci giochiamo, li guardiamo e studiamo, ce li imprimiamo nella mente.
Ero ad una di queste esercitazioni; a tempo scaduto eravamo rimasti in pochi, e un gruppetto di sole donne temerarie (Including me) to encircle the prof and his laptop. Someone asks what kind of material can be found on the Internet, prof says "everything" and to demonstrate, as a movie, it pulls all its various videos of dissections.
Crazy.
Not so much disgust: the impression is relative, for those not afraid of blood, bone, fat.
But at the moral and human is devastating.
see a body, a few days before was still a living person with a life and history, reduced to a mere heap of flesh, no dignity.
find yourself making these thoughts ... yes man - no man ... yes dignity - no dignity.
children, is tough, and quite a lot. This right is tough, because he wants to get you to the study of microscopic and in the meantime is likely to get away from the big picture.
I'll have to go through the dissection through the bodies of dead and lifeless, sooner or later.
But I promise to never lose the overview. The complexity as something more than the sum of its parts.
The feeling of humanity.
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